Panobinostat: first global approval

Area of Science:

• Oncology
• Pharmacology
• Molecular Biology

Background:

• Histone deacetylases (HDACs) regulate gene expression and chromatin structure, making them targets for cancer therapy.
• Panobinostat is a novel histone deacetylase (HDAC) inhibitor developed by Novartis.
• Dysregulation of HDACs is implicated in the pathogenesis of various cancers, including multiple myeloma.

Purpose of the Study:

• To summarize the key developmental milestones of panobinostat.
• To highlight the regulatory approvals and clinical development of panobinostat for cancer treatment.
• To provide an overview of panobinostat's mechanism of action as an HDAC inhibitor.

Main Methods:

• Review of preclinical and clinical data for panobinostat.
• Analysis of regulatory submission data for panobinostat.
• Summary of ongoing clinical trials investigating panobinostat in various tumor types.

Main Results:

• Oral panobinostat (Farydak®) is approved in the US in combination therapy for recurrent multiple myeloma.
• Regulatory submissions for panobinostat combination therapy in multiple myeloma have been made in the EU and Japan.
• Panobinostat is under investigation in diverse hematological and solid tumors.

Conclusions:

• Panobinostat represents a significant advancement in the treatment of multiple myeloma.
• The development of panobinostat highlights the therapeutic potential of HDAC inhibitors in oncology.
• Panobinostat's broad clinical development indicates its potential utility across a spectrum of cancers.